More than a month ahead of schedule, the FDA has approved Radicava (edaravone) for the treatment of amyotrophic lateral sclerosis (ALS).
In August 2016
, the Food and Drug Administration (FDA) first accepted filing of the drug, and RDR reported that a decision of the application was expected by June 16, 2017.
ALS, or "Lou Gehrig's Disease," is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. Motor neurons reach from the brain to the spinal cord and from the spinal cord to the muscles throughout the body.
The drug is already sold by Japanese pharmaceutical company Mitsubishi Tanabe Pharma Corp (MTPC) in both Japan and South Korea, but Friday’s approval marks the first such U.S. regulatory approval in more than 20 years.
"After learning about the use of edaravone to treat ALS in Japan, we rapidly engaged with the drug developer about filing a marketing application in the United States," said Eric Bastings, M.D., deputy director of the Division of Neurology Products in the FDA's Center for Drug Evaluation and Research. "This is the first new treatment approved by the FDA for ALS in many years, and we are pleased that people with ALS will now have an additional option."
Per a press release
, “the efficacy of edaravone for the treatment of ALS was demonstrated in a six-month clinical trial conducted in Japan. In the trial, 137 participants were randomized to receive edaravone or placebo. At Week 24, individuals receiving edaravone declined less on a clinical assessment of daily functioning compared to those receiving a placebo.”
The data was presented last year at the AAN Annual Meeting
, and showed the edaravone-treated group had a change in their ALS Functional Rating Scale-Revised (ALSFRS-R) score of -5.01 ± 0.64 compared to -7.50 ± 0.66 in the placebo group (P
= .001). The most common adverse events were contusion (16%), and dysphagia (13%).
The only other FDA-approved ALS treatment in the U.S., generic riluzole, has been proven to modestly slow the progression of the disease in some patients, but data showed the Radicava reduced the rate of functional decline in ALS patients by about a third, said VP of medical affairs, at MTPC's U.S. unit MT Pharma America (MTPA), Dr Jean Hubble.
The FDA granted this drug orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.